NCT02418455

Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age

An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less Than 5 Years Old

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 1 Day – 5 Years
• Healthy volunteers: Not accepted

Summary

The primary objective was to evaluate the effect of UX003 treatment in pediatric MPS VII participants less than 5 years of age on safety, tolerability, and efficacy as determined by the reduction of urinary glycosaminoglycans (uGAG) excretion.

Conditions

• Sly Syndrome
• MPS VII
• Mucopolysaccharidosis
• Mucopolysaccharidosis VII

Interventions

Type	Name	Description
DRUG	UX003	solution for intravenous infusion

Timeline

• Start date: 2015-07-21
• Primary completion: 2019-03-26
• Completion: 2019-03-26
• First posted: 2015-04-16
• Last updated: 2019-10-30
• Results posted: 2019-10-16

Locations

5 sites across 3 countries: United States, Portugal, Spain